First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

• Register: ClinicalTrials.gov
• Last refreshed on: 15 April 2024
• Main ID: NCT02675465
• Date of registration: 26/01/2016
• Prospective Registration: No
• Primary sponsor: Amicus Therapeutics
• Public title: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
• Scientific title: An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
• Date of first enrolment: January 2016
• Target sample size: 32
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02675465
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Australia, Germany, Netherlands, New Zealand, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Male and female subjects between 18 and 75years of age, inclusive

- Diagnosis of Pompe disease

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

- Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive

- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency
of once every other week

- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )

- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

ERT-experienced subjects (non-ambulatory):

- Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for =2 years

- Is wheelchair-bound

ERT-naïve subjects (ambulatory):

- Must be able to walk 200-500 meters on the 6MWT

- Has upright FVC must be 30% to 80% of predicted normal value

- Subject has never received alglucosidase alfa

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

- Has received ERT with alglucosidase alfa for >7years, inclusive

- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency
of once every other week

- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )

- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

Exclusion Criteria:

- Subject has received treatment with prohibited medications within 30 days of Baseline
Visit

- Subject, if female, is pregnant or breastfeeding at screening

- Subject, whether male or female, planning to conceive a child during the study

- Subject has a medical or any other extenuating condition or circumstance that may, in
opinion of investigator, pose an undue safety risk to the subject or compromise
his/her ability to comply with protocol requirements

- Subject has a history of allergy or sensitivity to miglustat or other iminosugars

- Subjects with active systemic autoimmune disease such as lupus, scleroderma, or
rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the
Amicus Medical Monitor

- Subjects with active bronchial asthma. All subjects with bronchial asthma must be
discussed with the Amicus Medical Monitor



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Drug: AT2221

Drug: ATB200

Primary Outcome(s)

Plasma GAA activity levels as measured by area under the plasma-drug concentration time curve. [Time Frame: 18 Weeks]

Plasma GAA activity levels as measured by time to reach the maximum observed plasma concentration (tmax). [Time Frame: 18 Weeks]

Plasma GAA activity levels as measured by maximum observed plasma concentration (Cmax). [Time Frame: 18 Weeks]

Safety and tolerability as measured by counts of Treatment Emergent Adverse Events (TEAEs), including Infusion Associated Reactions (IARs). [Time Frame: 18 weeks]

Secondary ID(s)

ATB200-02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors